Broadway’s biggest stars come together to raise money for Cystic Fibrosis

What do you get when Broadway’s biggest stars, such as Javier Muñoz and Gideon Glick, come together in the recording studio? One heck of a song. And one heck of a message.

Joined by Broadway veterans Laura Osnes, Christy Altomare and actress Sarah Levy, Muñoz and Glick have banded together for a new campaign — called the “Anyway” campaign — for an original song to help raise money for Emily’s Entourage.

At the center of the Entourage is Emily Kramer-Golinkoff: a 33-year-old daughter, sister and granddaughter who was diagnosed with Cystic Fibrosis when she was just a few weeks old. Her parents, Liza and Michael, have done everything in their power to raise Emily as if she was a normal child.

As the fatal disease only affects 70,000 people worldwide, funding for a cure is limited. Emily and her family are even more restrained by her specific mutation, which means medical funding is even rarer. And with a life expectancy of only 35-37 years for her kind of CF, time’s ticking.

Six years after the Kramer-Golinkoffs decided to take matters into their own hands, they’ve raised $3 million to drive high-impact research and speed up breakthroughs to research not only Emily’s mutation, but many other diseases including muscular dystrophy, inherited blood disorders and certain cancers.

They’ve also built a network of family, friends, and strangers from around the world, all of whom have been welcomed into the Entourage. Each Entourage member has been more inspired by Emily’s story than the next.

Take Elizabeth Phillipson-Weiner, from Emily’s hometown of Philadelphia, Pennsylvania and composer of “Anyway.” She took a simple journal entry from a songwriting retreat, turning lyrics like “when the going gets tough I ask questions” into a prolific melody.

Elizabeth explained to AOL Lifestyle. “The song wasn’t just cathartic for me, but could actually ring true for a lot of other people. I immediately thought of Emily.” Working with co-writers, producers and engineers, that melody was soon turned into a demo.

But as inspired as Elizabeth was by Emily’s story, the Entourage required “star power” to bring this project alive.

“I cold contacted agents and managers, I asked friends and friends of friends and friends of friends of friends,” said Elizabeth. “We did hear ‘no’ quite a bit, but whenever I became discouraged or frustrated I thought about who and what I was doing this for.”

This story was originally published on AOL.com

Cystic Fibrosis Awareness: Sharing A Story & The Facts

Cystic fibrosis is a devastating disease. About 30,000 children and adults have cystic fibrosis (CF) in The United States. While the life expectancy for someone with CF has doubled in the last 30 years, children and adults people still lose their lives to this disease every day. There is no cure yet. It’s time to find it.

How can you help? Use your voice and get involved! Participate in Great Strides walks and other events supporting CF and share the facts on social media and in your community. The Cystic Fibrosis Foundation is working hard to find a cure. Other foundations and organizations help the CF community as well. Like the CF Roundtable which supports adults with CF, there is a foundation that supports children and families living with CF: Claire’s Place Foundation.

Claire’s Place Foundation, founded by Claire Wineland, when she was just 14 years old, is a 501c3 non-profit organization designed to help children and families affected by cystic fibrosis. Claire was born with CF. In her short life, Claire has undergone more than 20 surgeries and has spent months at a time in the hospital.  She survived months in a coma on mechanical ventilation despite the odds and was also a Make-a-Wish recipient. She has 40% of ideal lung function and the doctors have estimated two years left to live. This is not, however, what characterizes or defines her. Claire has dedicated her life work to making a difference in the lives of others living with CF.

Now 21 years old as an adult living with CF, Claire’s Place Foundation is a way for Claire to give back with hope, strength, and joy.  She wants to live with intention and purpose. In her own words, “I know my life will be shorter so every day has meaning.”  She strives to find new ways to help the CF community and other children afflicted with chronic illness. Claire’s Place is making a huge impact through its two programs – The Extended Hospital Stay Fund and the Family Support Program.  These programs are in high demand and the foundation needs your donations to continue assisting young patients in need. You can meet Claire and support her foundation at her next fundraiser, “Glow Ride,” on August 18, 2018 in Hermosa Beach, CA.

To find others ways you can help bring awareness to CF find a local chapter near you.

Get involved. Donate. Come to events.

CF Foundation ‘Venture Philanthropy’ Model Crucial to CF Breakthroughs

By Larry Luxner

When the Cystic Fibrosis Foundation (CFF) was established in 1955, most people with cystic fibrosis (CF) didn’t make it to their sixth birthday. Today, the average life expectancy of a CF patient is 47 years.

To date, the U.S. Food and Drug Administration has approved 12 CF therapies. Three of them are CFTR modulators that treat the basic disease-causing defect, benefiting 60 percent of all patients, and more therapies are on the way.

Preston W. Campbell III, the CFF’s president and CEO, directly attributes this dramatic improvement to the foundation’s philosophy of “venture philanthropy.”

“We are now in Phase 3 CFTR trials that, if successful, will mean that as early as next year, more than 90 percent of all individuals with CF will have a highly effective therapy targeting CF’s basic defect,” he said. “More therapies that treat the complications of CF are in the pipeline than ever before.

“It begs the question: how did all of this happen?”

Campbell answered that during his March 26 presentation, “Patient advocates taking a real stand in drug development: How the CFF worked with biotech and pharma to find a cure,” at the 2018 World Orphan Drug Congress USA in Oxon Hill, Maryland.

Back in 1960, the Bethesda, Maryland-based foundation broke ground by establishing a Care Center Network to provide multidisciplinary care. Within five more years, it had formed a patient registry.

With only $400,000 in the bank, it would also commit $11 million to research, Campbell said. “Five years later, in 1985, the basic CF defect was identified, and in 1989, the CFTR gene was discovered. That opened the floodgates,” he added.

Campbell’s predecessor, Robert J. Beall, created the Therapeutics Development Program — now called its Venture Philanthropy Model — in 1998 to entice industry to focus on CF, and specifically on CFTR as a target. Its three components were financial assistance, research tools and scientific advice, and a clinical trials network.

“We would lower the risk for industry to come into the CF space. We also made our research tools and scientific advice freely available, and we also embedded the best scientists in the world in these industry programs,” said Campbell, who took over from Beall as head of the CFF in January 2016. “Finally, in order to make sure clinical trials were safely and efficiently done, we created a clinical trials network that originally had seven centers and now has 89.”

In the beginning, CFF’s investments were typically in the $1.5 million range. Ultimately, the foundation invested more than $100 million in Aurora and its successor, Vertex Pharmaceuticals, whose headquarters are in Boston.

To date, the FDA has approved three Vertex CFTR modulators: Kalydeco (ivacaftor) for patients with the G551D mutation in the CFTR gene (2012); Orkambi (lumacaftor/ivacaftor)for patients who are homozygous for F508del, the most common mutation in the CFTR gene (2015); and Symdeko (tezacaftor/ivacaftor) for homozygous F508del patients as well as others (2018).

“Payments are milestone-based, so we pay for success,” Campbell said. “A scientific advisory committee determines if milestones are met and if the project should continue. Successful programs offer a return on our investment, so if the program is foundering, we shake hands and walk away.”

To continue to full article, please click here.

SIX Ways to PAY IT FORWARD to CF ROUNDTABLE!

By Jeanie Hanley, President

Greetings CF Roundtable Subscriber!

May is CF Awareness month. What better way to “Pay It Forward” than by supporting CF Roundtable which has been vital to the CF community! Please consider making a tax-deductible donation today.

This is YOUR CF Roundtable and because of your generosity, YOU have made it possible for nearly 30 years. 100% of your donation goes into the newsletter and many outreach programs. All work is done by volunteers with CF like Andrea, our Executive Editor, whose inspirational words regarding her 18 years of transplant are below:

Eighteen Years of Life Post-Transplant

By Andrea Eisenman, Executive Editor of CF Roundtable

Reflecting back on my life for the last 18 years post-transplant, I am amazed I have lived so long. Way longer than I expected, considering the 50 percent median survival of 5 years after a bilateral lung transplant. I am grateful for this time in which I was able to get married, go back to school for various interests like film and cooking, and care for my mom in her later years, share my life with people I care about and never in recent memory felt this good.

While I have enjoyed a good quality of life, it came with a price of total compliance almost to the point of being neurotic at times (my doctors probably get sick of my calls and emails), a daily exercise regimen and lots of rest. But I found that if I did things I enjoyed like tennis, pickle ball or swimming, it helped get the exercise for that day done while it was fun and social.

I have been extremely fortunate as not only do I have this longevity with transplant and I feel pretty well. Aside from the last 12 months, I have had the ability to travel and do most things my peers do. While I had some setbacks recently, I am starting to feel better. I keep a positive outlook and do what is needed. I can see how precious this gift of life is and I hope that when my time comes to be a donor, the person who gets my organs enjoys them as much as I enjoyed these lungs.

DONATE LIFE!

Please consider Paying It Forward in these six ways:

 

  • Unrestricted Gifts – your contribution will go to the program that needs it most.
  • Milestone Celebration: for a transplant anniversary, birth of a child, wedding, or a birthday. There is no greater reward than celebrating YOU and YOUR accomplishments.
  • Tribute Gifts – donate in honor or in memory of someone.  
  • USACFA Endowment Fund – consider contributing which will get CF Roundtable closer to be self-sustaining forever! Please contact us if you are able to contribute.
  • Matching Gifts – if your employer has this program, then let us know!
  • Bequest – A simple and easy way to remember CF Roundtable in your estate planning.  To establish a bequest, please contact us.

 

To make a donation, click here DONATE NOW!

Or MAIL a check USACFA

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Contact us at cfroundtable@usacfa.org for any further assistance.

USACFA proudly publishes CF Roundtable and all its associated programs; USACFA is a 501(c)(3) nonprofit organization. All donations are tax-deductible.

Thank you!

Boomer Esiason Wants You… to Run the TCS NYC Marathon!

NOVEMBER 5, 2017
STEP 1: REGISTER
To run for Team Boomer

STEP 2: INTERVIEW
with Team Boomer staffer Katie

STEP 3: RUN NEW YORK CITY! Continue reading Boomer Esiason Wants You… to Run the TCS NYC Marathon!

USACFA’s Fall 2017 Lauren Melissa Kelly Scholarship Winners

The US Adult CF Association (USACFA) is excited to announce our recipients of the Lauren Melissa Kelly Scholarship: Congratulations to Jacob Greene and Elizabeth Shea! They will be awarded $2500 each. Continue reading USACFA’s Fall 2017 Lauren Melissa Kelly Scholarship Winners

Team Boomer Asks, Are You In?

Run with Team Boomer in the TCS New York City Marathon

NOVEMBER 5, 2017
STEP 1: REGISTER
To run for Team Boomer

STEP 2: INTERVIEW
with Team Boomer staffer Katie

STEP 3: RUN NEW YORK CITY! Continue reading Team Boomer Asks, Are You In?

Maxing Out Your Strides – Mark A. Levine

By Mark Levine

Here we are again, on the precipice of another Great Strides Walk season. Not sure about you but I have already sent out my second email, a gentle reminder, to my entire list to increase the size of my team and boost my donations. It’s an exciting time of the year and a great event to be a part of that fulfills a lot in my Continue reading Maxing Out Your Strides – Mark A. Levine